| First Author | Tinklenberg JA | Year | 2019 |
| Journal | J Neuropathol Exp Neurol | Volume | 78 |
| Issue | 2 | Pages | 130-139 |
| PubMed ID | 30597051 | Mgi Jnum | J:340943 |
| Mgi Id | MGI:7531260 | Doi | 10.1093/jnen/nly120 |
| Citation | Tinklenberg JA, et al. (2019) Myostatin Inhibition Using ActRIIB-mFc Does Not Produce Weight Gain or Strength in the Nebulin Conditional KO Mouse. J Neuropathol Exp Neurol 78(2):130-139 |
| abstractText | Mutations in at least 12 genes are responsible for a group of congenital skeletal muscle diseases known as nemaline myopathies (NMs). NMs are associated with a range of clinical symptoms and pathological changes often including the presence of cytoplasmic rod-like structures (nemaline bodies) and myofiber hypotrophy. Our recent work has identified a variable degree of behavioral benefit when treating 2 NM mouse models due to mutations in Acta1 with myostatin inhibition. This study is focused on the effects of delivering ActRIIB-mFc (Acceleron; a myostatin inhibitor) to the nebulin conditional knockout KO (Neb cKO) mouse model of NM. Treatment of Neb cKO mice with ActRIIB-mFc did not produce increases in weight gain, strength, myofiber size, or hypertrophic pathway signaling. Overall, our studies demonstrate a lack of response in Neb cKO mice to myostatin inhibition, which differs from the response observed when treating other NM models. |
